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Pain Management in Children and Young Adults With Sickle Cell Disease

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ClinicalTrials.gov Identifier: NCT01954927
Recruitment Status : Completed
First Posted : October 7, 2013
Last Update Posted : July 6, 2018
Sponsor:
Collaborator:
Scan | Design Foundation
Information provided by (Responsible Party):
St. Jude Children's Research Hospital

Brief Summary:

This is a phase II double-blind placebo-controlled clinical trial evaluating the effect of gabapentin when added to standard pain management for patients with sickle cell disease experiencing acute pain crisis in the ambulatory care setting.

Sickle cell pain is different for every patient. Some patients get complete relief from routine pain medicines, and others need more time or more doses of pain medicines before the pain goes away completely. It is known that humans have many types of pain, including something called neuropathic pain. Neuropathic pain in other conditions (such as diabetes) has been treated successfully with a medicine called gabapentin. The investigators in this study suspect that some sickle cell pain is a combination of pain types. They would like to see if adding gabapentin to the usual pain medicines makes pain go away faster or more completely.

Primary Objective:

  • To assess the analgesic efficacy of gabapentin vs. placebo for pain during vaso-occlusive crisis (VOC) in participants with sickle cell disease (SCD). A response to study drug will be defined by a decrease in pain score of ≥ 33% between presentation to the acute care setting and assessment at 3 hours post administration of study drug.

Secondary Objective:

  • To compare the total morphine equivalent dose (mg/kg) used to control pain during VOC between presentation to the acute care setting and assessment at 3 hours post administration of study drug in the gabapentin vs. placebo groups.

Condition or disease Intervention/treatment Phase
Sickle Cell Disease Drug: Gabapentin Drug: Placebo Phase 2

Detailed Description:

Upon participant enrollment, study staff will randomize the participant to one of 2 possible treatment arms: a single dose of gabapentin or a single dose of placebo. Morphine or other opioid and non-steroidal anti-inflammatory drugs will be available to both groups as needed for pain and will be administered according to the current standard of care for pain in VOC from the Department of Hematology at St. Jude Children's Research Hospital (SJCRH). Randomization will be performed in the SJCRH pharmacy by a pharmacist. The randomization will be stratified by three age categories (1-3 years of age, 4-6 years, and 7 years or older) for which distinct pain assessment tools are applied and for 2 pain score categories at assessment at presentation (4-6 and 7-10, respectively). A block randomization with block sizes varying randomly between 4 and 6 will be used in each stratum.

Pain scores will be obtained at presentation to the acute care setting and 3 hours (± 15 minutes) post administration of study drug. Participants who were discharged will be contacted by study staff between 24 and 72 hours following administration of study drug to see if there have been any side effects. Patients who were admitted after administration of the study drug will be monitored through hospital record to determine if any unexpected events occurred. After this follow up, participation in the study is complete.


Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 90 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Pain Management of Vaso-Occlusive Crisis in Children and Young Adults With Sickle Cell Disease
Actual Study Start Date : October 7, 2013
Actual Primary Completion Date : January 3, 2018
Actual Study Completion Date : January 3, 2018


Arm Intervention/treatment
Active Comparator: Gabapentin
Patients will be randomized to receive one dose of gabapentin.
Drug: Gabapentin
Gabapentin is supplied as an oral suspension. Patients randomized to the gabapentin arm will receive a single dose of gabapentin as soon after enrollment as feasible. The gabapentin dose will be given orally and will be approximately 15 mg/kg with a maximum dose of 900mg.
Other Name: Neurontin(R)

Placebo Comparator: Placebo
Patients will be randomized to receive one dose of placebo.
Drug: Placebo
Placebo will be prepared by the SJCRH pharmacy and will be similar in appearance, quantity and taste to the gabapentin drug. Patients randomized to the placebo arm will receive a single dose of placebo as soon after enrollment as feasible. The placebo dose will be given orally and will be approximately 15 mg/kg with a maximum dose of 900mg.




Primary Outcome Measures :
  1. Number and proportion of participants with successful pain interventions by arm [ Time Frame: Baseline and 3 hours post administration of study drug ]
    Pain scales used are the numerical rating system, the Faces Pain Scale, and the FLACC pain scale (for patients 7 years or older, ages 4-6 years, or less than 4 years, respectively). For each patient, if the reduction of the pain scores (0=no pain and 10=worst possible pain) between presentation to the acute care setting and 3 hours post administration of study drug is 33% or greater, then this patient will be defined as having a successful intervention. The proportions of successful interventions in the gabapentin and placebo groups will be estimated and compared using Z-test.


Secondary Outcome Measures :
  1. Total dose (mg) of opioid analgesic administered [ Time Frame: All doses given between baseline and 3 hours post administration of study drug ]
    Summary statistics of the total morphine dose or morphine equivalent (mg/kg) used to control pain during VOC between presentation to the acute care setting and 3 hours post administration of study drug, in the gabapentin and placebo groups will be provided. Test of normality such as Shapiro-Wilk test will be applied to the total morphine dose or morphine equivalent (mg/kg) to examine their deviation from the normal distribution. A two-sample t-test or Wilcoxon rank sum test will be used to compare the total morphine dose or morphine equivalent (mg/kg) for the gabapentin vs. placebo groups depending on whether the normality assumption of the data holds.



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Ages Eligible for Study:   1 Year to 20 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Participant must have sickle cell disease (any genotype) documented in the St. Jude medical record.
  • Participant must be seeking care for acute vaso-occlusive pain at St. Jude Children's Research Hospital.
  • Participant age must be ≥1 year and <21 years.

Exclusion Criteria:

  • Prior randomization in this study.
  • Mild pain (score <4) or pain for which treatment with opioid is not indicated.
  • Pregnant or lactating female.
  • Decreased glomerular filtration rate (GFT) (<60ml/min/1.73m^2) as estimated by the revised Schwartz equation.
  • Current treatment with gabapentinoid drugs (gabapentin or pregabalin).
  • Known seizure disorder.
  • Current treatment with antiepileptic agents.
  • Pain in combination with other clinical symptoms that require additional interventions, including fever with focus, acute chest syndrome, acute injury, or splenic sequestration.
  • Allergy to gabapentin.
  • Current participation in another research study with an investigational new drug/device (IND/IDE) agent.
  • Inability or unwillingness of research participant or legal guardian/representative to give written informed consent.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01954927


Locations
United States, Tennessee
St. Jude Children's Research Hospital
Memphis, Tennessee, United States, 38105
Sponsors and Collaborators
St. Jude Children's Research Hospital
Scan | Design Foundation
Investigators
Principal Investigator: Doralina Anghelescu, MD St. Jude Children's Research Hospital

Additional Information:
Responsible Party: St. Jude Children's Research Hospital
ClinicalTrials.gov Identifier: NCT01954927     History of Changes
Other Study ID Numbers: PMVOC
First Posted: October 7, 2013    Key Record Dates
Last Update Posted: July 6, 2018
Last Verified: July 2018

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by St. Jude Children's Research Hospital:
Pain control

Additional relevant MeSH terms:
Anemia, Sickle Cell
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn
Gabapentin
gamma-Aminobutyric Acid
Analgesics
Sensory System Agents
Peripheral Nervous System Agents
Physiological Effects of Drugs
Anticonvulsants
Antiparkinson Agents
Anti-Dyskinesia Agents
Calcium Channel Blockers
Membrane Transport Modulators
Molecular Mechanisms of Pharmacological Action
Anti-Anxiety Agents
Tranquilizing Agents
Central Nervous System Depressants
Psychotropic Drugs
Excitatory Amino Acid Antagonists
Excitatory Amino Acid Agents
Neurotransmitter Agents
Antimanic Agents
GABA Agents